Custom Cytosine Base Editor (AAV)
Custom Cytosine Base Editor (AAV)
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Precision C-to-T base editing via dual-AAV split-intein delivery for in vivo applications. SpCas9-based CBE systems reconstitute in target cells from co-transduced N-terminal and C-terminal AAV vectors. Ideal for liver, muscle, and CNS applications requiring C-to-T editing with established SpCas9 PAM compatibility and editing windows. Choose BE3 for foundational validation or BE4max for enhanced efficiency and reduced indels.
- BE3 Base Editor: First cytosine base editor enabling C-to-T conversion at protospacer positions 4–8 without double-strand DNA breaks. Achieves ~30% editing efficiency with ~1.1% indels in human cells using APOBEC1 deaminase fused to Cas9 D10A nickase. Essential reference for benchmarking against optimized editors (BE4max, ABEmax) and for studying CBE mechanism and constraints.
- bGH PolyA Signal: Compact (225 bp) polyadenylation signal from bovine growth hormone with 3-fold higher expression than SV40 early polyA in key mammalian cell types. Size advantage over SV40 polyA makes it the preferred terminator for AAV vectors with limited cargo space. Standard terminator in commercial gene therapy and lentiviral vector platforms with decades of in vivo validation.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
- BE4max Base Editor: Optimized cytosine base editor with 1.8× higher efficiency than BE4. Enhanced nuclear import and codon optimization boost C-to-T conversion.
- bGH PolyA Signal: Compact (225 bp) polyadenylation signal from bovine growth hormone with 3-fold higher expression than SV40 early polyA in key mammalian cell types. Size advantage over SV40 polyA makes it the preferred terminator for AAV vectors with limited cargo space. Standard terminator in commercial gene therapy and lentiviral vector platforms with decades of in vivo validation.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
- AncBE4max Base Editor: Advanced CBE using ancestral APOBEC reconstruction. Minimized transcriptome-wide off-targets with narrower editing window and lower toxicity.
- bGH PolyA Signal: Compact (225 bp) polyadenylation signal from bovine growth hormone with 3-fold higher expression than SV40 early polyA in key mammalian cell types. Size advantage over SV40 polyA makes it the preferred terminator for AAV vectors with limited cargo space. Standard terminator in commercial gene therapy and lentiviral vector platforms with decades of in vivo validation.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
- Target-AID Base Editor: Alternative cytosine base editor using sea lamprey cytidine deaminase. Narrower editing window (positions 2-4) with reduced cellular toxicity.
- bGH PolyA Signal: Compact (225 bp) polyadenylation signal from bovine growth hormone with 3-fold higher expression than SV40 early polyA in key mammalian cell types. Size advantage over SV40 polyA makes it the preferred terminator for AAV vectors with limited cargo space. Standard terminator in commercial gene therapy and lentiviral vector platforms with decades of in vivo validation.
- U6 Promoter: RNA polymerase III promoter that drives constitutive expression of small RNAs. Provides strong, stable transcription with precise transcriptional start and termination sites.
Deliverables
- Titer: 1e13 VG/mL or higher
- Full Capsid Ratio: 0.85 or higher
- Delivery Timeline: 18–21 days
Specifications
Single stranded AAV produced in 293T cells and purified through two (2) rounds of CsCl ultracentrifugation and stored in 0.001% F-68 in DPBS with additional 150 mM NaCl. Ships on dry ice overnight.
This construct can be packaged in hundreds of AAV serotypes to target your specific tissue. We offer all standard serotypes (AAV1-9) plus specialized variants for muscle (MyoAAV-2A, MyoAAV-1A, AAVMyo), brain (PHP.eB, AAV9P31, AAV2-retro), and clinical applications (AAV2, Spark100). Other popular options include AAV-rh.74, AAV6, AAV6.2FF, and AAV3. Browse our complete serotype library or contact us for recommendations.
References
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